EpiBio Seminar Series | Anna Heath

"Bayesian Methods to Improve Clinical Trials for Children"

Presented by Dr. Anna Heath an Associate Professor at the Biostatistics Division, Dalla Lana School of Public Health, University of Toronto as a part of the EpiBio Seminar Series 2026/27.

Attend in person: PHFM 3015
Attend online: please request the Zoom link at epibio@uwo.ca 

Abstract: Enrolling children into clinical trials can be challenging due to the relative rarity of diseases, ethical challenges with enrolling children into trials and lower willingness to engage in research for children and their families. Thus, many interventions that are currently used in practice have not been tested appropriately in children. In fact, around 80% of medications used in children’s hospitals are used off-label, meaning that they have not been approved for that indication. Bayesian clinical trials can be more flexible than standard clinical trial designs and they can incorporate external information to reduce sample size requirements. As such, novel Bayesian designs can be developed to address these key challenges in paediatric clinical trials and provide feasible clinical trials to evaluate medical interventions for use in children.
This presentation will focus on two different designs that are currently being used to test interventions in children. Each design uses an alternative approach to improve the feasibility of these clinical trials. Firstly, we will discuss a novel method to rank interventions during the trial to determine the best intervention. This method is particularly useful when it is challenging to specify a “control” intervention as there is a great variation in current practice, which is common in paediatrics. We will then discuss a design that uses Bayesian hierarchical modelling to evaluate interventions when sample size is severely limited, e.g., for rare diseases, by combining information across rare and common diagnoses.